Infants and young children with severe atopic dermatitis (AD) have a high burden of disease with a strong impact on quality of life. Here we assess long-term efficacy and safety of dupilumab in pediatric patients aged 6 months to 5 years with severe AD. This is a subgroup analysis of patients aged 6 months to 5 years enrolled in the ongoing LIBERTY AD PED open-label extension (OLE) study of dupilumab who had previously participated in the parent study LIBERTY AD PRESCHOOL part B and had severe AD (Investigator's Global Assessment [IGA] = 4) at parent study baseline. Patients received weight-tiered dupilumab every 4 weeks (200 mg for patients weighing 5 to < 15 kg; 300 mg for patients weighing 15 to < 30 kg). Key endpoints included the incidence and rate of treatment-emergent adverse events (TEAEs), the proportion of patients with a ≥ 75% improvement in Eczema Area and Severity Index (EASI-75) from parent study baseline, proportions of patients achieving IGA = 0/1 and IGA ≤ 2, and the proportion of patients with a ≥ 6-point improvement in Children's Dermatology Life Quality Index (CDLQI) for patients aged ≥ 4 years or Infants' Dermatitis Quality of Life (IDQoL) questionnaire for patients aged < 4 years. This analysis included 121 patients, of whom 50 completed the week 104 visit. TEAEs were reported in 88% of patients; most TEAEs were mild or moderate and not related to treatment. Common TEAEs included upper respiratory tract infection, nasopharyngitis, and cough. Conjunctivitis events were reported in 19% of patients and were mild or moderate, with a median duration of 8 days. No conjunctivitis event led to treatment discontinuation, and most events resolved during the study. One drug-related event (severe urticaria) led to treatment discontinuation, but it was not serious and resolved over time. Serious TEAEs were reported in 17 patients (14%), including one drug-related pinworm event. No serious TEAE led to treatment discontinuation. By week 4 of the OLE study, patients who had received placebo in the parent study exhibited efficacy improvements comparable to patients who had received dupilumab. By week 104, 96% of patients achieved EASI-75 from parent study baseline, 27% of patients achieved an IGA score of 0/1 (clear/almost clear skin), 92% of patients achieved IGA ≤ 2 (clear skin to mild AD), and the least squares mean percent change in EASI from parent study baseline was - 89%. Additionally, 89% (23/26) of patients achieved a ≥ 6-point (clinically meaningful) improvement in CDLQI, and 100% (3/3) of patients achieved a ≥ 6-point improvement in IDQoL. Long-term treatment with dupilumab for up to 2 years showed acceptable safety and sustained efficacy in signs, symptoms, and quality of life in patients aged 6 months to 5 years with severe AD, with a rapid improvement for patients who had received placebo in the parent study. Long-term safety in the OLE study was consistent with the short-term safety profile observed in the parent study, with no new safety signals detected. [Graphical abstract and plain language summary available] CLINICAL TRIAL REGISTRATION: ClinicalTrials.gov Identifiers: NCT02612454 and NCT03346434 (part B). Atopic dermatitis (AD) is one of the most common inflammatory diseases in children. Severe AD can have a strong negative impact on children’s well-being, with a high risk of long-term persistence. In a previous study, treatment with dupilumab for 16 weeks demonstrated significant health benefits in children aged 6 months to 5 years with severe AD. To analyze the effects of dupilumab treatment for a longer time, children who had participated in the 16-week study continued in the present study, in which they received 200/300 mg of dupilumab (depending on body weight) every 4 weeks for up to 2 years. During 2 years of treatment, 88% of patients reported health issues, mostly mild or moderate and not related to dupilumab. Only one patient interrupted treatment due to a health issue (intense skin rash) that was considered a side effect of dupilumab, but it was not considered serious and resolved over time. In another patient, one serious pinworm infection was also considered related to dupilumab, but it did not cause the patient to interrupt treatment. By the end of the 2 years, 27% of patients achieved clear or almost clear skin, 92% had clear skin to mild AD, and 96% achieved a 75% improvement in AD extent and severity. Most patients also reported improvements in quality of life. In summary, long-term treatment with dupilumab was beneficial for infants and young children with severe AD with a low risk of side effects, consistent with results observed in older children, adolescents, and adults.
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arXiv · 2026-04-20
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